Trial Information

Summary: A Postmarketing Observational Study to Assess the Efficacy and Safety of Factor VIII/von Willebrand Factor Complex (Human), Alphanate® in Preventing Excessive Bleeding During Surgery in Subjects with Congenital Type 3 von Willebrand Disease

von Willebrand Disease is a bleeding problem in which the patient has a problem with von Willebrand Factor, a protein in the blood which helps with clot formation. von Willebrand Disease can be a partial lack, as in Type 1, or a severe or complete lack as in Type 3. Alphanate is purified von Willebrand Factor which is collected from human blood. Alphanate also contains Factor VIII, another protein in the blood involved in clotting. Human serum from many blood donors are mixed, or pooled, into one batch. The Alphanate components are taken from this pool of serum and treated in several different ways to kill any and all infectious parts of the serum. Alphanate is very pure, and in early clinical trials has proven to be very safe.

Subject Inclusion Criteria
In order to qualify to receive treatment in the study, each patient must satisfy all criteria listed below:

• The subject is a male or female 7 years of age or older as determined by subject’s medical records.
• The subject has been diagnosed of inherited Type 3 VWD as determined by subject’s medical records.
• The subject needs a surgical procedure (at least 10 surgical procedures have to be considered as “Major” according to Appendix I).
• The subject is expected to respond to exogenously administered FVIII/VWF according to Investigator’s judgment.
• The subject freely gives written informed consent. Patients who are not legally permitted to provide written consent must sign a form of assent for study participation, and written consent must be provided by a parent or legal guardian.

Subject Exclusion Criteria
Patients will be excluded if any of the following exclusion criteria are met:

• The subject has been diagnosed of Type 1, Type 2 or acquired VWD as determined by subject’s medical records.
• The subject is known to have history of intolerance to any Alphanate® containing substance.
• The subject is known to have history of anaphylactic reaction(s) to blood or blood components.
• Liver function tests (AST, ALT, bilirubin) > 2.5 x upper limit of normal (ULN).
• Renal function test (creatinine, BUN) > 1.5 x ULN.
• The subject is known or suspected to have present or past inhibitor activity (antibodies) directed against FVIII or VWF.
• The subject is known to abuse alcohol or illicit drug use within the past 12 months.
• The subject is participating in another clinical study involving an investigational treatment, or participated within the past 4 weeks. Studies consisting of data and blood sampling collections on a regular or long-term basis are exempt from this exclusion.
• The subject is unlikely to adhere to the protocol requirements of the study.

If you would like to learn more about participating in this study, please send an e-mail message using the form below.